The Final Pillar of GGC’s Precision Medicine Initiative: ACTION
As we’ve explored in previous posts, the Greenwood Genetic Center’s Precision Medicine Initiative (PMI) is designed to address the many challenges faced by patients with genetic disorders. Through our focus on Access, Analysis, and Answers, we’ve been working tirelessly to improve how genetic care is delivered. Today, we’re excited to complete this series of posts by diving into the final and most impactful pillar: ACTION.
At GGC, Action means getting patients the treatment or therapy they need for their genetic conditions—now. This is not just about finding answers but ensuring those answers lead to real, effective treatments that improve lives. Here’s how we’re turning research into results.
The ultimate goal of the Action pillar is clear: to connect patients with treatments that can directly improve or even cure their conditions. But making this a reality is complex. It involves clinical trials, drug repurposing, and collaboration. Here’s what we’re doing at GGC to ensure that every patient receives the most personalized, timely care possible:
1. Clinical Trials for Precision Treatments
One of our first steps in bringing action to our patients is through clinical trials—with a focus on n-of-1 trials, which are tailored specifically for rare, individualized conditions. These trials provide treatments designed precisely for each patient, giving us the flexibility to approach each genetic disorder uniquely.
2. Expanding Collaboration with MUSC
With our partners at the Medical University of South Carolina (MUSC), we are aligning efforts to offer genetic therapeutics and more treatment options to patients. From developing biomarkers for genetic disorders to matching patients with the right clinical trials, this collaboration ensures that our patients are connected with the most promising treatment options available.
And through the MUSC Precision Health Institute (PHI), with GGC’s President and CEO, Dr. Steve Skinner serving as the clinical lead, we are ensuring that discoveries—like new genetic findings and gene therapy options—are quickly translated into real treatments.
3. Repurposing Drugs for Immediate Impact
Developing new drugs typically takes years, but we know our patients can’t wait that long. That’s why GGC is focusing on drug repurposing—testing already FDA-approved drugs for new uses. We’re particularly excited about the potential of over-the-counter supplements and existing medications to bring immediate relief to patients with rare genetic conditions, with recent funding for these projects from Gene Spotlight and Cure Sanfilippo Foundation. The Genomic Discovery Program (GDP) plays a central role in this process, connecting patients with both existing therapies and potential new treatments.
4. Curry Chair
GGC’s Ravenel Boykin Curry Chair in Translational Genomics and Therapeutics, Dr. Mike Lyons, has dedicated his career to improving patient care in the field of genetic medicine, and now, as the head of the Action arm of GGC’s Precision Medicine Initiative, he is spearheading efforts to translate scientific discoveries into life-changing treatments for our patients. Under his leadership, GGC will continue to bridge the gap between research and clinical care, bringing the best possible outcomes for every patient.
5. Metabolic Treatment Program: Immediate Action, Lifelong Impact
One of the most impactful aspects of our Action pillar that has already made significant strides is our Metabolic Treatment Program, led by Dr. Emily Black. GGC’s Metabolic Treatment Program provides immediate care for patients with rare metabolic genetic disorders, many of which are diagnosed through newborn screening. These conditions can be life-threatening, but with timely intervention, patients can lead healthy, fulfilling lives.
From genetic counseling to enzyme replacement to dietary management, our team is dedicated to providing comprehensive care that prevents disabilities and saves lives. Through telehealth, we’re expanding access to these services, ensuring that all patients, no matter where they are, can benefit from the care they need.
6. Pioneering Alzheimer’s Research
In a major expansion of our research efforts, GGC’s Director of Research, Dr. Rich Steet, led GGC’s launch of the Carroll A. Campbell, Jr. Alzheimer’s Initiative in 2024, which focuses on groundbreaking research into mitochondrial dysfunction and its role in Alzheimer’s disease and other neurological disorders. In collaboration with MitoSense and the U.S. Department of Veterans Affairs, GGC is pioneering the use of Mitochondria Organelle Transplantation (MOTTM) to potentially restore and improve function in brain cells.
This research could have profound implications for not only Alzheimer’s patients but also for children with mitochondrial disorders. With the BioSpherix Xvivo System, GGC is one of the few centers in the country with the technology to isolate mitochondria for clinical trials. And as we move closer to those trials, we are one step closer to turning hope into treatment.
Moving Forward: The Work Continues
As we conclude our four-part series on the Precision Medicine Initiative, it’s important to remember that Action is not the final step in our journey—it is the beginning of a new era of genetic care. Every patient we serve is at the heart of everything we do. Whether it’s through clinical trials, drug repurposing, or expanding access to care, we are committed to delivering personalized, cutting-edge treatments that can transform lives today.
We’re grateful to everyone who has supported GGC’s mission and the Therapeutic Research & Development Fund. With your help, we are making the future of genetic medicine brighter, faster, and more accessible for every patient.
Thank you for following along with our series on the Precision Medicine Initiative. The journey continues, and together, we will make a lasting impact on the lives of those affected by genetic disorders.
Want to support ACTION at GGC?
Your investment in the GGC’s Precision Medicine Initiative helps bring answers, hope, and treatments to patients with rare diseases. Learn more at ggc.org/foundation.